Stem cell researchers tout potential for sickle cell disease cure

Editor's Note

A novel stem cell transplant treatment for sickle cell disease (SCD) led to disease-free outcomes for most participants in a clinical trial at a lower cost than gene therapy, according to a February 27 report in Sickle Cell Disease News. The approach, which relies on a less intensive conditioning regimen and expands donor eligibility, resulted in 88% of patients remaining free of disease-related clinical events after two years.

As detailed in the article, SCD occurs when genetic mutations cause red blood cells to adopt a sickle shape, leading to blockages, pain crises, and progressive organ damage. Traditional bone marrow transplants require a fully matched donor to minimize the risk of graft-versus-host disease (GVHD), a severe complication where donor cells attack the recipient’s body. The new method, known as haploidentical bone marrow transplantation, allows for donors who are only half-matched, typically a family member, which increases the likelihood of finding a suitable match.

The phase 2 trial followed 42 adults with severe SCD for a median of 37 months. According to Sickle Cell Disease News, findings indicate  that 95% of participants were alive after two years, and 88% had no disease-related events—outcomes comparable to or better than gene therapy,. However, three patients experienced graft failure, and 22.4% developed chronic GVHD within the first two years. Four participants died, including two due to early infections, but researchers noted these complications were expected for the procedure. Findings were published in The New England Journal of Medicine Evidence.